A Study of the Safety and Efficacy of Fabrazyme in Patients With Fabry Disease
Public ClinicalTrials.gov record NCT00074971. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Multi-center, Open-Label Extension Study of the Safety and Efficacy of Recombinant Human a-Galactosidase A (r-haGAL) Replacement in Patients With Fabry Disease
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
People with Fabry disease have an alteration in their genetic material (DNA) which causes a deficiency of the a-galactosidase A enzyme. Fabrazyme is a drug that helps to breakdown and remove certain types of fatty substances called "glycolipids." These glycolipids are normally present within the body in most cells. In Fabry disease, glycolipids build up in various tissues such as the liver, kidney, skin, and blood vessels because a-galactosidase A is not present, or is present in small quantities. The build up of glycolipid ("globatriaosylceramide" or "GL-3") levels in these tissues in particular is thought to cause the clinical symptoms that are common to Fabry disease. This study will test the safety and efficacy of Fabrazyme in the treatment of patients with Fabry disease.
Study identification
- NCT ID
- NCT00074971
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 3
- Enrollment
- 58 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 16 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Oct 1999 (Type not available)
- Primary completion
- Not listed
- Completion
- Dec 2004 (Type not available)
- Last update posted
- Dec 4, 2013
1999 – 2004
United States locations
- U.S. sites
- 14
- U.S. states
- 12
- U.S. cities
- 14
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Cedars-Sinai Medical Center | Los Angeles | California | 90048 | — |
| University of California San Fransisco | San Francisco | California | 94143 | — |
| Northwest Oncology & Hematology Associates | Coral Springs | Florida | 33065 | — |
| Children's Memorial Hospital | Chicago | Illinois | 60614 | — |
| University of Iowa Hospital and Clinics | Iowa City | Iowa | 52242 | — |
| Greater Baltimore Medical Center | Baltimore | Maryland | 21204 | — |
| Beth Israel Deaconess Medical Center | Boston | Massachusetts | 02215 | — |
| Fetal Diagnostic and Imaging Center | Billings | Montana | 59101 | — |
| University of New Mexico | Albuquerque | New Mexico | 87131 | — |
| Mount Sinai School of Medicine | New York | New York | 10029 | — |
| University of Rochester School of Medicine | Rochester | New York | 14642 | — |
| Hart Family Practice | Hickory | North Carolina | 28601 | — |
| Hematology/Oncology Associates of South Texas | San Antonio | Texas | 78229 | — |
| University of Washington School of Medicine | Seattle | Washington | 98195 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 6 non-U.S. sites.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT00074971, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Dec 4, 2013 · Synced Oct 3, 2026
Related: full search, browse by condition, browse by drug or therapy, browse by sponsor, browse by U.S. city.
Open the official record
The complete public registry record, listed eligibility criteria, and available contact information for NCT00074971 live on ClinicalTrials.gov.